Last week,
the ASBMB Careers Blog featured education and research
jobs focused on rare diseases in observance of Rare
Disease Day. Another key area of research and development in the
rare-disease field is orphan-drug discovery. The U.S. Food and Drug Administration
defines
an orphan drug as “those intended for the safe and effective
treatment, diagnosis or prevention of rare diseases/disorders that affect fewer
than 200,000 people in the U.S., or that affect more than 200,000 persons but
are not expected to recover the costs of developing and marketing a treatment
drug.”

To date, only
an estimated
5 percent of rare diseases have an associated FDA-approved treatment.
Fortunately, this is changing and the orphan-drug market is projected to grow
for a number of reasons. This includes the adoption of legislation (e.g., Orphan
Drug Act) that provides financial incentives to and streamlines the
drug-approval process for pharmaceutical companies. Advances in science and
technology like gene therapy also are making treatment options more viable
because the majority of rare diseases have a genetic basis. Additionally, patient-advocacy
groups are making the voices of the patient community heard and raising
awareness of the need for more resources to help fight rare diseases.

This week,
we bring you resources to learn more about finding career opportunities in
orphan-drug discovery and a selection of related industry jobs. Note that many of
the companies listed in the jobs roundup have multiple openings across their R&D
and ancillary (e.g., biomanufacturing, medical writing, regulatory affairs)
departments for bachelor’s to Ph.D. degree holders. Be sure to check out the
respective career sites for each company to view even more job opportunities.

Orphan-drug discovery resources

The
FDA’s Office of Orphan Products
Development maintains the Orphan Drug
Designations and Approvals database that contains information on
drugs and biologics that have been submitted and/or approved for orphan-drug
designation, including the name of the company that developed the drug. You also
can learn more about the orphan-drug designation process and related resources
on the OOPD
website.

If
you are interested in pursuing a career in orphan-drug discovery, it is
important to learn more about the drug-development process and to stay updated
on trends in the field. Here are few resources that may help you do so.

Global
Genes, a nonprofit that advocates for the rare-disease community, offers a free
online course in understanding drug
development that reviews the key stages of drug development and the
critical role of patients in this process.

Industry reports and trade
publications can
be a good source of information to learn more about ongoing orphan-drug development
efforts. For example, McKinsey and Company publishes insights on the
pharmaceutical and medical industry, which includes a recent article on how
to successfully launch a rare-disease drug. Pharmaceutical Research
and Manufacturers of America is another organization that reports on
pharmaceutical-industry trends. Check out the PhRMA website From Hope to Cures
for a collection of relevant publications on medicines in development for rare
diseases. Also, EvaluatePharma publishes an informative orphan-drug
report.

Weekly jobs roundup

The
pharmaceutical company Boehringer
Ingelheim is seeking a summer
intern in operations to joins its Idiopathic Pulmonary Fibrosis department
located in Ridgefield, Conn. The intern will gain experience working with a
specialty-care, orphan-drug department and assist with operational tasks (e.g.,
communication projects, meeting coordination). Minimum qualifications include current
status as an undergraduate, graduate or professional student in good academic
standing and a major related to the internship. No application deadline is
provided.

The
global biopharmaceutical company Celgene
is hiring a senior
medical writer within its Medical Writing and Submission Planning
department located in Seattle. The writer will assist with the review and
preparation of clinical and regulatory documents that include applications for
orphan-drug designations. Minimum qualifications include a bachelor’s degree in
a science and three to five years of regulatory writing or related experience.
No application deadline is provided.

Precision
Medicine Group, a
contract research organization for pharmaceutical and life-sciences clients,
has multiple
positions open within its Oncology and Rare Disease division. These
positions include multiple remote/home-based positions as clinical
research/trial associates and project managers/directors to oversee the proper
design, setup and monitoring of clinical studies for clients. See the job
postings for details on qualifications. No application deadlines are provided.

AveXis, Inc., a clinical-stage, gene-therapy
company dedicated to developing novel treatments for rare neurological genetic
diseases, is hiring a scientist
in research and development at its San Diego location. The incumbent
will conduct in vivo experiments (e.g., animal-dosing studies) for existing and
pipeline therapies. Minimum qualifications include a bachelor’s degree in
biology, neuroscience, animal science or a related field and prior laboratory
experience that includes working with mice. No application deadline is
provided.

Forbius, a clinical-stage,
protein-engineering company that develops biotherapeutics for cancer and
fibrotic diseases, is seeking a research
associate in preclinical research to support experiments in the
areas of immune oncology and orphan diseases and manage the laboratory. The
position could be located in Austin or Montreal. Minimum qualifications include
a bachelor’s degree in a biological discipline with seven years of experience
or a master’s degree and three years of experience. No application deadline is
provided.

Adverum Biotechnologies (Menlo Park, Calif.), a
clinical-stage, gene-therapy company targeting unmet medical needs in
ophthalmology and rare diseases, is in search of an associate scientist in
assay development. The scientist will develop quantitative
molecular-biology and cell-based assays in support of pharmacokinetics,
pharmacodynamics and product release. Minimum qualifications include a
bachelor’s degree in a biological science plus four to five years of experience
in a biotech company or a master’s degree and two to three years of experience
(or an equivalent combination of education and experience). No application
deadline is provided.

PTC Therapeutics, a biopharmaceutical company
developing small-molecule drugs that target post-transcriptional control
processes for the treatment of orphan disorders, is hiring a biology
research associate in the neurodegenerative disease program at its
South Plainfield, N.J. location. The research associate with support preclinical
research studies to identify and screen activity of new drug entities. Minimum
qualifications include a master’s degree in a biological discipline and two
years of related experience. No application deadline is provided.

Ultragenyx, a biopharmaceutical company
committed to developing treatments for rare and ultra-rare diseases, is seeking
a scientist
in mRNA process development to support the company’s nucleic-acid
therapy pipeline. The scientist will be responsible for setting up a new lab to
enable small-scale mRNA production at its facility located in Brisbane, Calif.
Minimum qualifications include a Ph.D. in a life-sciences field with a focus on
molecular biology and two years of relevant working experience, a master’s
degree with six years of experience or a bachelor’s degree with eight years of
experience. No application deadline is provided.

Rubius Therapeutics (Cambridge, Mass.), a
biopharmaceutical company pioneering a new era of cellular medicines for the
treatment of rare diseases, cancer and autoimmune diseases, has an opening for
a scientist/associate
scientist in protein chemistry. The scientist will develop novel
strategies for protein/cellular/nanoparticle conjugations using click-chemistry
reactions and/or incorporation of unnatural amino acids. Minimum qualifications
include a Ph.D. and related research experience in biology, biochemistry,
biophysics, chemistry or a related field (or a bachelor’s or master’s degree
and five to 15 years of experience).

Editor’s note

The ASBMB
regularly hosts webinars in career and professional-development areas. The next
webinar is this Thurs., Feb. 21, at 1 p.m. Eastern time on the topic of responsible
conduct of research. Join Suzanne Barbour, dean of the University of
Georgia Graduate School, as she discusses ways scientists can conduct research
in a safe and ethical manner. Also, be sure to check out the ASBMB website
for other upcoming webinars and to view archived topics.

Donna Kridelbaugh is a contributor to the
ASBMB Careers Blog. She holds an advanced degree in microbiology and is a
former lab manager.

Stay updated on new posts by following theASBMB on social media or
click “follow” on this blog (must be a member and signed in). Also, be sure to
check out the ASBMB Job Board for
even more job listings.

Feb. 28
marks Rare
Disease Day. It is estimated that one in 10 Americans lives with a
rare (sometimes called orphan) disease. A disease is considered rare in the
U.S. if it affects less than 200,000 people, and there are about 7,000 rare
diseases classified to date. For some quick stats about rare diseases, see Global Genes’
Rare Facts.

While
these diseases may be deemed rare, in many ways, they create more of a disease burden
on patients and their families who may have a harder time in attaining an
accurate diagnosis, accessing treatment options and getting coverage of
healthcare and related expenses. Funding for disease research and drug
development also can be a challenge due to basic economics, whereas there is
less of a demand for treatment because there are fewer patients.

This Rare
Disease Day, the ASBMB has gathered a collection of
personal and research stories to raise awareness of this important
health observance. You also can share snippets of your own research or personal
experiences with rare diseases on social media using the hashtag #RareDiseaseDay.

There are
a number of career opportunities to get involved with rare diseases, from basic
research into disease mechanisms to advocating for more funding and resources
to develop treatment options. This week, we feature some rare-disease resources
that can help you find potential employers, along with a weekly jobs roundup. An
upcoming blog post will highlight jobs in orphan drug discovery to advance
therapies for rare diseases.

Rare disease resources

The
National Center for Advancing Translational Sciences and NIH Clinical Center
are sponsoring Rare Disease Day at
NIH on Feb. 28 at the main campus in Bethesda, Md. The event
highlights research collaborations ongoing to address rare diseases with topical
speakers, posters and exhibits, and networking opportunities. Admission is free
and open to the public. There also is a live
webcast available for those who cannot attend in person, and you can
follow along on Twitter using the hashtag #RDDNIH.

There
are a number of rare-disease databases
that list out associated information, such as clinical resources, patient-support
groups and research news. This information can be helpful to learn who all is
active in the advocacy and research landscapes for specific rare diseases. You then
can look more into these groups for potential job and training opportunities.
Here are a few of these databases:

Another
way to locate potential researchers to work with is to identify research
centers and networks focused on rare diseases. As an example, the Rare Diseases Clinical Research Network
is an initiative of the NCATS Office of Rare Diseases Research. The RDCRN
website lists the currently funded research groups, along with the diseases
each focus on and ongoing studies.

Clinicaltrials.gov can be used to search for current
and past clinical trials related to rare-disease therapies. A search within the
field of “condition or disease” for “rare
diseases” yields 446 results. The search results provided relevant
information on the sponsors of each study and related publications, which can
be useful information for your job searches.

Weekly jobs roundup

The
National Organization for Rare Disorders
offers internship opportunities
for undergraduate and graduate students in various areas (e.g., public policy
and advocacy, education and research) at its multiple office locations. The
deadline to apply for summer internships is May 1. See the website for more
details.

Global Genes (Aliso Viejo, Calif., or remote)
is hiring a program manager to
produce quality content and organize speakers for its educational conferences
and resources targeted towards the rare-disease patient and advocate
communities. Events include the annual RARE Patient Advocacy Summit and RARE Drug
Development Symposium. Minimum qualifications include a bachelor’s degree in journalism,
public health or life science and two to three years of science-communications
experience. No application deadline is provided.

ICF, a global consulting and
technology services provider, is recruiting an information
specialist for the NIH’s Genetic and Rare Diseases Information
Center. The specialist will respond to public inquiries about rare and genetic
disorders and share relevant resources and information. The position can be
based in Rockville, Md., or remote. Minimum qualifications include a master’s
degree in human genetics or genetic counseling, one year of experience in
clinical genetics and preference for candidates who are bilingual in English
and Spanish. No application deadline is provided.

The
Neuroendocrine Tumor Research Foundation
(Boston, Mass.) is seeking a director of research to lead
its scientific research program to find treatments and cures for rare
neuroendocrine tumors. Responsibilities include managing the research grant
programs, liaising with the advisory boards and organizing the annual research
symposium. Minimum qualifications include a master’s degree in a related field
and five years of relevant medical or research experience. No application
deadline is provided.

Boston Children’s Hospital has an opening for a post-bachelor’s
research
assistant in neurobiology to support the Human Neuron Core research
group. The RA will be involved with disease-modeling, differentiation and
phenotyping assays. This includes processing clinical samples from patients
with rare neurological disorders. Minimum qualifications include a bachelor’s
degree in biology, molecular biology, biochemistry, neurobiology or related field
and experience with cell cultures. No application deadline is provided.

The
Department of Pediatrics and Division of Medical Genetics at the University of Pittsburgh School of Medicine,
in conjunction with the Center for Rare Disease Therapy at the Children's
Hospital of Pittsburgh, is seeking an open-rank, tenure-track faculty
member to lead a basic research program in rare diseases using
cutting-edge technologies. See the job posting for details on how to apply. No
application deadline is provided.

The National Institute of Dental
and Craniofacial Research
is seeking a staff scientist
to research rare skeletal diseases. Minimum qualifications
include a Ph.D. with experience in bone and mineral metabolism and skeletal
stem cell research. The application deadline is Feb. 28.

Additionally,
the Gourh lab within the National
Institute of Arthritis and Musculoskeletal and Skin Diseases is hiring two postdoctoral
researchers in immunology/molecular
biology and bioinformatics/genetics
to assist with studies on the genetics of the rare autoimmune disease called systemic
sclerosis (scleroderma) among African-American patients. See the postings for
details. No application deadlines are provided.

Bonus opportunity

The NIH Academy
Enrichment Program is accepting applications from recent college
graduates of diverse backgrounds who are interested in gaining biomedical
research experience while learning more about addressing health
disparities. Participants spend a year or two conducting research
with an NIH mentor and participate in academy activities to explore the role of
science, policy and community engagement in eliminating health disparities.
Applicants must be U.S. citizens or permanent residents, eligible for the NIH
Postbac IRTA program and planning to apply for graduate school after the
program. You must both apply to the NIH Postbac IRTA program and submit a
letter of interest to the NAEP to be considered. See the website for
application instructions and links. The application deadline is Feb. 26.

Donna Kridelbaugh is a contributor to the ASBMB Careers Blog. She holds an advanced degree in microbiology and is a former lab manager.

Stay updated on new posts by following theASBMB on social media or click “follow” on this blog (must be a member and signed in). Also, be sure to check out the ASBMB Job Board for even more job listings.

This
shutdown has further amplified the climate of science-funding uncertainty and
underscores the critical need for scientists to be actively involved in science
policy and advocacy. You have to make sure your voice is heard to ensure
adequate resources are in place to support the important work you do.

If the
latest government shutdown has you motivated to take action, the ASBMB public
affairs team has many opportunities for early-career scientists to get engaged
in science advocacy. One training event for graduate students and postdoctoral
researchers is the annual ASBMB
Capitol Hill Day with applications due by Feb. 10.

For those
of you interested in pursuing a lifelong career in the policy realm, this week
we feature a roundup of science-policy jobs and training opportunities to help
you make that transition.

Weekly jobs roundup

The
American Association for Cancer Research
is accepting applications for its two-year science
policy fellowship program. Fellows gain policy experience working rotations
within the U.S. Congress, AACR Office of Science Policy and Government Affairs and
National Cancer Institute or U.S. Food and Drug Administration. Minimum
qualifications include a terminal science degree received within the past five
years and legal eligibility to work in the U.S. The application deadline is
Feb. 20.

The
American Cancer Society Cancer Action
Network (Washington, D.C.) is recruiting a senior
policy analyst to support the organization’s efforts in
evidence-based policy and legislative solutions that will eliminate cancer as a
major health problem. This position focuses on drug/device development and
approval, federal appropriations, clinical trials and access to care and
prevention issues. Minimum qualifications include a bachelor’s or master’s
degree with a five to seven years of related work experience. No application
deadline is provided. (Note: The ACS CAN also hosts policy
interns who are senior undergraduates or graduate students. See the
posting for more details.)

The
American Society of Human Genetics
(Rockville, Md.) is hiring a policy and advocacy
manager to monitor policy issues, draft related policy statements
and materials and promote member engagement in advocacy campaigns. Minimum
qualifications include a bachelor’s degree in a relevant science or policy area
and four years of experience with science or health policy. No application
deadline is provided.

The
Board on Health Sciences Policy within the National
Academies of Sciences, Engineering and Medicine (Washington, D.C.) has
several open positions that include a senior
program assistant and program officer. The HSP works to strengthen the rigor of biomedical
research and ensure the translatability of research to policy-making. As such,
the board considers the ethical, legal and social contexts of scientific and
technologic advances. Check out the job listings for more details. No
application deadlines are provided.

The
global healthcare company Novo Nordisk
hosts summer
interns in federal government affairs. Interns will work with its
government affairs team in Washington, D.C., to conduct outreach to
congressional offices on sponsorship of priority legislation and help support
the annual Novo Nordisk Inc. Capitol Hill Advocacy Day. Minimum qualifications
include enrollment in a bachelor’s or master’s degree program in political
science, health policy, government, public health or a related discipline. No
application deadline is provided.

The
DuPont Specialty Products Division is
looking for a political
action committee manager for its government affairs office located in
Washington, D.C. The incumbent will manage the daily operations of the office,
provide administrative support to the team and perform related support tasks
(e.g., track congressional hearings, prepare lobbying reports). Minimum
qualifications include a bachelor’s degree and a strong desire to transition
into government affairs. No application deadline is provided.

The
biopharmaceutical company Regeneron
is hiring several congressional
liaisons to work in Washington, D.C., to advance its public-policy
and legislative initiatives. Minimum qualifications include a bachelor’s degree
and six years of experience in a policy capacity, such as working with federal legislators
and regulators. No application deadline is provided.

Author’s note

If you
need to gain more experience in science policy, consider forming your own
science-policy group to work on issues that matter to your peer network. Check
out this past Scientific
American blog post by Thaiesha Wright
with some good tips on how to do so. For funding such a startup, you can look
into establishing the group as an official student organization and applying
for mini-grants through professional societies and organizations. As an
example, the National Science
Policy Network is accepting applications for its second round of
microgrants. The application deadline is Feb. 22.

Donna Kridelbaugh is a contributor to the ASBMB Careers Blog. She holds an advanced degree in microbiology and is a former lab manager.

Stay updated on new posts by following theASBMB on social media or click “follow” on this blog (must be a member and signed in). Also, be sure to check out the ASBMB Job Board for even more job listings.